Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States
Keywords: AE, Adverse Event; SAE, Serious Adverse Event; CF, Cystic Fibrosis; CFF, Cystic Fibrosis Foundation; CFTR, Cystic Fibrosis Transmembrane Conductance Regulator; FDA, Federal Drug Administration; FEV1, Forced expiratory volume in 1 sCystic fibrosis (CF); Cy